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. 2009 Apr 21;17(7):1136–1144. doi: 10.1038/mt.2009.87

Figure 4.

Figure 4

Correction of phenotypic characteristics in NOD/SCID MPS I mice. Mice treated with 25 µg transposon plasmids +SB were evaluated 18 weeks postinjection and compared to untreated, age-matched controls from the same mouse colony. (a) Correction of hepatomegaly in treated NOD/SCID MPS I mice. For evaluation of hepatomegaly, the entire carcass and the intact liver were weighed. The ratio of liver weight to total body weight in normal controls was 5.7 ± 0.2% (in females, n = 9) and 5.2 ± 0.3% (in males, n = 3). In MPS I mice the respective ratios were: 6.6 ± 0.1% (in females, n = 3) and 7.2 ± 0.3% (in males). Treated MPS I female and male mice had respective ratios of 5.1 ± 0.3% (n = 6) and 5.3 ± 0.1% (n = 7), which is significantly lower than in MPS I mice (P = 0.007 and P = 0.0002 in females and males, respectively). The values for males and females in each group were in the same range and therefore were pooled. Gray diamonds, untreated, normal (WT and heterozygous) controls; black open diamonds, treated NOD/SCID MPS I mice; black filled diamonds, untreated NOD/SCID MPS I mice. (b) Representative skull microradiographs from male mice. Untreated normal (heterozygous) control (left), treated MPS I (center), and untreated MPS I (right). The white arrows indicate increases in thickness of cranial zygomatic arches in the untreated MPS I mouse. Measurements are given in Table 3. (c) Representative sections of bone marrow (1–3) and synovium (4–6) from untreated normal (WT and heterozygous) control (left), treated MPS I (center), and untreated MPS I (right) animals. Arrows in panel 3 indicate clusters of foamy macrophages in the untreated MPS I mouse; these cells were absent in mice in the other two groups. Arrows in panels 4–6 indicate synovial membrane, which is normal in untreated normal (left) and treated MPS I (center) animals and is expanded by the presence of large numbers of foamy macrophages in the untreated MPS I animal (right). Hematoxylin and eosin stain. Bar = 50 µm (sections 1–3) and 100 µm (sections 4–6). MPS I, mucopolysaccharidosis type I; NOD/SCID, nonobese diabetic/severe combined immunodeficiency; SB, Sleeping Beauty; WT, wild-type.